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Molecular Diagnosis & Therapy|February 1, 2019
Gene Therapy for Beta-Hemoglobinopathies: Milestones, New Therapies and ChallengesValentina Ghiaccio, Maxwell Chappell, Stefano Rivella, et al.
Journal of Virological Methods|February 5, 2023
Protocol for a high titer of BaEV-Rless pseudotyped lentiviral vector: Focus on syncytium formation and detachmentKazuhiro Noguchi, Yasuhiro Ikawa, Mika Takenaka, et al.
Biorxiv : the Preprint Server for Biology|April 1, 2024
Effective Gene Therapy for Metachromatic Leukodystrophy Achieved with Minimal Lentiviral Genomic IntegrationsLucas Tricoli, Sunetra Sase, Julia Hacker, et al.
Haematologica|May 23, 2020
2'-O-methoxyethyl splice-switching oligos correct splicing from IVS2-745 β-thalassemia patient cells restoring HbA production and chain rebalanceAlisa Dong, Valentina Ghiaccio, Irene Motta, et al.
Molecular Therapy. Nucleic Acids|April 2, 2025
Effective gene therapy for metachromatic leukodystrophy achieved with minimal lentiviral genomic integrationsLucas Tricoli, Sunetra Sase, Julia L Hacker, et al.
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